Improving how a disease is treated doesn’t always require a scientific breakthrough. It can be as straightforward as identifying which medical centers already have the healthiest patients, finding out exactly what they do to ensure their patients’ health, and getting other clinics to use those methods. Such “quality improvement” projects have already made a big difference in the lives of people with cystic fibrosis and cardiac disease. The Enroll-HD Care Improvement Committee (CIC), an international group of researchers involved with the study, is now beginning to apply these principles to HD. The goal: “To create a world where the care of current patients is simply better,” says Martha Nance, MD, one of the co-chairs of the CIC.

The basic idea is straightforward. Every HD center has their own preferred ways of treating the symptoms of the disease—for example, tending to prescribe one psychiatric drug over another, or recommending every patient (or none) to a particular type of physical therapy. Some of these methods are undoubtedly more effective at treating symptoms or keeping people healthier than others, but without hard data on who does better, it’s impossible to tell.

The only way to know for sure is to compare the results from many people at many different centers. Because Enroll-HD is collecting data from clinics around the world, it’s an opportunity to make these broad comparisons across different regions and identify “best practices”—the treatment choices that on average lead to the healthiest, best-functioning patients.

It may be surprising that this hasn’t been done before, but because HD is an “orphan” disease, meaning that it affects relatively few people, few studies have been able to compare current practices to see which are most effective. Experienced HD doctors rely on their knowledge and best judgment in treating their patients, but not all doctors who treat HD have this experience, and even the best instincts can be misleading. “We really don’t know what aspects of what we do are actually important,” says Nance, who is director of the HDSA center of excellence at Hennepin County Medical Center. “We have no idea whether one antidepressant works better than another in terms of treating depression—or maybe no antidepressant works better.” The data collected within Enroll-HD can answer some of these questions.

Learning from cystic fibrosis and Parkinson’s disease

Identifying best practices can make a big difference in the lives of people with chronic disease. A quality improvement project in cystic fibrosis (CF) has increased the lifespan of people by 10 years since 2002. “Just by putting forward these standards of care, and organizing the care teams according to best practices, they were able to actually increase life expectancy,” says Joe Giuliano, CHDI director of clinical operations. “That’s ultimately what we’re all about, to improve the lives of people with HD and their families.”

The goal: To create a world where the care of current patients is simply better.

A similar effort launched in 2009, the Parkinson’s Outcome Project, has found “dramatic variation” in how well people do with the disease at different clinics that specialize in Parkinson’s disease, says Peter Schmidt, PhD, the vice president of research and professional programs for the National Parkinson Foundation, who is in charge of the project. “In medicine, there’s an assumption that any one physician could be exchanged for another—that the most important thing is which pills patients are given, and what they’re told to do,” says Schmidt. “We rarely consider the performance of a physician,” or take into account the way that a center’s staff works together to ensure that patients get all the services they need.

But in fact the data collected for the Parkinson’s project show that the doctor and the care site account for about 25 percent of the differences in health and quality of life in PD patients. This project has already identified a way of organizing health care that makes a big difference in depression, one common problem in PD.

Improving HD Care

In HD the first step will be to see what is actually being done now. The committee is surveying active Enroll-HD sites to establish basic facts for each center: what services they offer, who is on the HD care team, how often patients are seen, and how the clinic is organized. “It will give us some understanding of the context of care,” says Jan Frich, MD, PhD, of the University of Oslo and currently a visiting professor at the Yale School of Public Health.

Meanwhile, a working group is starting a small-scale analysis of data from REGISTRY (the long-term study that preceded Enroll-HD in Europe) to begin identifying which medical practices might be most relevant to improving quality of life—the first step in zeroing in on what really makes a difference. “We’d like to learn more about how the model of care may actually have an impact on the patients and family,” says Frich.

Getting the best treatment can have a big impact on mood, energy, and ordinary life activities

In cystic fibrosis, lung function and body weight are easy to measure and clear signs of how well a patient is doing, but HD does not have any such obvious benchmarks yet. “In some patient groups it’s easier to identify the relevant measures, but it’s a huge challenge in HD. But I think it’s worth trying,” says Frich. “HD is a complex disease with many symptoms, and my guess is that we’d need several indicators rather than just one.” Candidates include measures of motor function, behavioral symptoms, or possibly body mass index (weight), but the main focus will be the overall wellbeing of the patient and family.

To begin with, one possible yardstick to compare different practices and centers will be patient quality of life, which can be measured with a symptom check-list. Obviously, quality of life is affected by more than just medical care, but getting the best treatment can have a big impact on mood, energy, and ordinary life activities.

The promise of quality improvement projects is being able to make a big difference in health care now, even before any great new drugs are invented. In PD, says Schmidt, the numbers suggest that excellent neurological care is equivalent to a drug that reduces the impact of the disease by 15 percent—potentially a big difference in many people’s lives. Meanwhile, the challenge in quality improvement projects is figuring out which aspects of treatment make a big difference. A lot of measurements are required to tease apart which parts of care are the most important. It’s also important to have a wide range of practices to compare. Because Enroll-HD is a large international study, it has both of these ingredients. “The strength of Enroll-HD is having the numbers to ask these kinds of questions,” says Nance. “The power is in the numbers.”